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Hope for Patients with a Lethal Form of Leukemia

Posted by andreahgreif in The LLS Blog, 12 September 2014 · 4,067 views

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An important study that could transform the outlook for a group of patients with a rare but lethal subset of acute lymphoblastic leukemia (ALL) appeared this week in the New England Journal of Medicine (subscription require to read full article). A commentary, “A Call to Action for Acute Lymphoblastic Leukemia” (subscription required) by Timothy A. Graubert, M.D. (also an LLS-grant recipient) also accompanies the study.

The research leading to this study (Targetable Kinase-Activating Lesions in Ph-like Acute Lymphoblastic Leukemia) was substantially funded by grants from The Leukemia & Lymphoma Society and could guide future research in the area of personalized medicine.

The researchers, from St. Jude Children’s Research Hospital, National Instutites of Health, Univeristy of Colorado, University of New Mexico and others, focus on patients with a subset of ALL called Philadelphia (Ph) chromosome-like ALL. While long-term survival for younger pediatric patients with ALL has improved to nearly 90 percent, young adult patients with ALL are more prone to fall into this subset that comes with a very poor prognosis.

The researchers used various genomic approaches to identify the genetic alterations associated with Ph-like ALL. They found that approximately 11.9 percent of children ages 1-15 have the Ph-like ALL, but that the percentage rose with age, with approximately 27 percent young adults, aged 21-39, falling into this high-risk subset.

The researchers also reported on results of eight patients with Ph chromosome-like ALL being treated with existing drugs called tyrosine kinase inhibitors (TKIs), which are commonly used to successfully treat patients with chronic myeloid leukemia and other types of blood cancers. Each patient experienced a dramatic immediate benefit when the TKI was added to their treatment regimen, and five patients remain in remission with no detectable cancer, one for more than a year. While seven out of eight patients in this small study were children, the findings pave the way for clinical trials involving patients regardless of age.

Dr. Graubert notes in his commentary that the approach taken by the doctors on the study, tailoring treatment based on molecular profiling, could provide a paradigm for treating patients with other high-risk cancers.



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